Risdiplam CAS No. 1825352-65-5 Pharmaceutical Raw Material
overview
Risdiplam is a potent, oral SMN2 (survival of motor neuron 2) splicing modifier and core pharmaceutical API. It specifically modulates SMN2 gene splicing to increase functional SMN protein production, approved for treating spinal muscular atrophy (SMA).
Our products comply with strict international standards to ensure high purity, stable efficacy, and reliable supply for clinical use and biomedical research.
Physical and Chemical Properties
- Density: 1.50±0.1 g/cm³ (Predicted, 20 °C)
- pKa: 9.41±0.20 (Predicted, 25 °C)
- Solubility: Soluble in DMSO
1. Unique Mechanism of Action and Strong Targeting
It is the world's first approved small-molecule drug that directly acts on RNA as a target. It promotes the production of functional SMN protein through dual-site specific regulation of the splicing process of the SMN2 gene. Its target is more precise, which can fundamentally make up for the insufficient protein expression caused by the deletion or mutation of the SMN1 gene.
2. Excellent Systemic Distribution Effect
It has the ability to cross the blood-brain barrier and can act on both the central nervous system and peripheral tissues to comprehensively increase the level of SMN protein in the body. It has a more comprehensive therapeutic effect on spinal muscular atrophy and can better improve the motor function of patients.
3. Wide Applicable Population Range
It is suitable for patients with spinal muscular atrophy aged 2 months and above, covering the entire age group from infants to adults, including patients with different severity types.
It is mainly used for the treatment of spinal muscular atrophy (SMA), a hereditary motor neuron disease caused by the deletion or mutation of the SMN1 gene. The specific applicable scenarios include:
1. Treating patients with various types of spinal muscular atrophy aged 2 months and above, including severe type I (onset in infancy with low median survival rate), moderate type II (able to sit but unable to stand) and mild type III (may lose walking ability in adulthood).
2. Delaying the progression of muscle weakness and atrophy in patients, improving motor function and enhancing the quality of life of patients by increasing the level of functional SMN protein in the body.
We implement strict full-process quality control, with each batch of Risdiplam undergoing rigorous testing of key indicators including purity, solubility, stability, and biological activity to meet standards.
Equipped with a stable raw material supply chain, we ensure continuous large-scale supply. Our professional technical team provides tailored services, including formulation optimization, application guidance, and post-sales technical support to address clinical and research needs.
As a pioneering SMN2 splicing modifier, Risdiplam has transformed the treatment of spinal muscular atrophy, offering a convenient and effective oral therapy for patients of all ages. With its targeted mechanism, broad efficacy, exceptional quality, and reliable supply capacity, we are committed to providing high-quality Risdiplam API and professional services for global pharmaceutical companies, research institutions, and clinical applications.
Choose our Risdiplam to meet your needs in pharmaceutical formulation, SMA research, and clinical treatment. Contact us for more product details and cooperation support.